Primary pulmonary hypertension has a long-standing reputation as a rare but progressive disease that is particularly difficult to treat. Although current treatment options include vasodilators, anticoagulants, or transplantation (lung or heart-lung), they have not been shown to improve survival in a prospective, randomized study. There is some hope, however, in the form of a continuous intravenous infusion with prostacyclin (epoprostenol).

In a study in a February issue of the New England Journal of Medicine, researchers at P&S and at 13 other sites that make up the Primary Pulmonary Hypertension Study Group investigated the effects of 12 weeks of continuous infusion of epoprostenol combined with conventional therapy on 41 patients with severe primary pulmonary hypertension, compared with 40 patients on conventional therapy alone. Dr. Robyn J. Barst, associate professor of clinical pediatrics, directed the study.

The researchers found that, compared with patients undergoing conventional therapy alone, those on epoprostenol plus conventional therapy showed an improvement in exercise capacity, quality of life, and clinical and hemodynamic measures. In addition, the epoprostenol significantly improved survival compared with conventional therapy alone.
This is not the first study that has shown promising results for epoprostenol. Clinical research on the drug dates back to 1987, when Dr. Barst began using epoprostenol to treat children with severe primary pulmonary hypertension. Delivered by continuous intravenous infusion using a portable pump, the drug slowed or even reversed the course of the disease in several children. The success of those early treatments led to the founding of the Children's Pulmonary Hypertension Center, the first of its kind in the country.